WHAT IS THE CADTH REIMBURSEMENT RECOMMENDATION FOR AMVUTTRA?: CADTH recommends that Amvuttra be reimbursed by public drug plans for the treatment of stage 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin-mediated (hATTR) amyloidosis if certain conditions are met. WHICH PATIENTS ARE ELIGIBLE FOR COVERAGE? Amvuttra should only be covered to treat adults with stage 1 or stage 2 genetically confirmed hATTR amyloidosis with polyneuropathy (hATTR-PN) who are symptomatic with early-stage neuropathy, do not have severe heart failure symptoms, and have not had a liver transplant. A patient’s response to treatment with Amvuttra should be assessed at least every 6 months to determine whether they would benefit from continued treatment. Treatment with Amvuttra should not be continued in patients who are permanently bedridden and dependent on assistance for basic activities of daily living or who are receiving end-of-life care. WHAT ARE THE CONDITIONS FOR REIMBURSEMENT? Amvuttra should only be reimbursed if the patient is under the care of a specialist with experience in the diagnosis and management of hATTR-PN and should not be reimbursed if it is used in combination with interfering ribonucleic acid drugs or transthyretin stabilizers. The cost of Amvuttra should be reduced so that it does not cost more than other drugs for hATTR amyloidosis. WHY DID CADTH MAKE THIS RECOMMENDATION? Evidence from a clinical trial showed that, in patients with hATTR-PN, treatment with Amvuttra improved neuropathy-related neurologic function and quality of life, and reduced disability due to neuropathy, when compared to treatment with placebo. In addition, the trial’s results suggested that Amvuttra had similar efficacy and safety as the currently available treatment option, patisiran. Amvuttra provides a subcutaneous drug option with less frequent administration that can be administered in a patient’s home, which addresses a need identified by patients. Based on CADTH’s assessment of the health economic evidence, Amvuttra does not represent good value to the health care system at the public list price. The committee determined that there is not enough evidence to justify a greater cost for Amvuttra compared with the least costly treatment reimbursed for hATTR-PN. Based on public list prices, Amvuttra is estimated to cost the public drug plans approximately $23.8 million over the next 3 years.
WHAT IS HATTR AMYLOIDOSIS? hATTR amyloidosis is caused by alterations in a gene that makes a protein called TTR. As a result of this genetic alteration, an abnormal protein called amyloid builds up in the body’s organs and peripheral nerves causing organs to not function properly, as well as nerve damage. In patients with hATTR-PN, amyloids primarily build up in the peripheral nerves. hATTR amyloidosis is considered a rare disease, affecting about 10,000 people worldwide. UNMET NEEDS IN HATTR AMYLOIDOSIS: Patients with hATTR-PN need effective treatments that slow disease progression, have a low risk of adverse events (AEs), provide a convenient route of administration, and have infrequent dosing. HOW MUCH DOES AMVUTTRA COST? Treatment with Amvuttra is expected to cost approximately $572,164 per patient per year.
